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Vol. LX, No. 21
October 17, 2008

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  Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a gene transfer procedure.  
  Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a gene transfer procedure.  

Promising Trial for Blinding Disease

Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a gene transfer procedure in an NEI-funded, phase 1 clinical trial. Participants in the study had one genetic form of Leber congenital amaurosis (LCA) caused by mutations in a specific gene and they all had visual impairment from birth. They received a subretinal injection to replace the nonfunctioning gene in preselected regions of the retina with less degeneration of photoreceptor cells. During the 90-day period of the study, the vision in all three young adults was partially restored. The study was the first to show that gene therapy can improve both day and night vision in patients with LCA. The findings, which included no reports of adverse effects from the therapy, were published online in the Sept. 22 issue of Proceedings of the National Academy of Sciences and in the Sept. 7 issue of Human Gene Therapy. The new reports extend findings of two other papers published earlier this year in the New England Journal of Medicine.

A New Model for Cystic Fibrosis

In a study funded in part by NHLBI and NIDDK, researchers for the first time used a genetically altered animal model for cystic fibrosis (CF) that closely matches the characteristics of the disease in humans. Using a pig model, scientists can now better understand how the complications of the complex, multi-organ disease develop. This could lead to new avenues for research in prevention and treatment. CF—an inherited disease of the mucus-secreting glands—affects multiple organs including the lungs, pancreas, liver, intestines and sinuses. The disease causes mucus to become thick and sticky and to build up in the lungs and in the pancreas, blocking airways, disrupting the digestive system and resulting in recurrent, destructive infections and digestive problems. Before this study, published in the Sept. 26 issue of Science, mice had been the only animal model for CF. But mice don’t exhibit typical symptoms of the disease in humans, so finding a better model was crucial to furthering research.

Searching for Better Ways to Control Asthma

According to a study funded by NIAID, monitoring levels of exhaled nitric oxide in adolescents with asthma and adjusting treatment accordingly doesn’t improve the course of the disease. The study, conducted by the Inner City Asthma Consortium and reported in the Sept. 20 issue of The Lancet, looked at approximately 550 adolescents in 10 cities across the country. The idea behind the research was to determine whether this kind of measurement would allow better control of the disease when used in addition to treatments based on national guidelines developed at NIH. It was the largest study to date testing exhaled nitric oxide as a biomarker for asthma management. Asthma affects about 9 percent of children under age 17 in the U.S. and its causes are still unknown. A biomarker of airway inflammation could be a useful clinical tool for gauging medical needs in asthma patients.

Dietary Supplements No Better than Placebo For Osteoarthritis

A 2-year study funded by NCCAM and NIAMS has shown that the popular dietary supplements glucosamine and chondroitin sulfate— used together or alone—did not appear to fare better than sugar pills in slowing loss of cartilage in osteoarthritis of the knee. However, the study results are difficult to interpret because participants taking the sugar pills had smaller loss of cartilage than scientists had predicted. The findings, published in the October issue of Arthritis & Rheumatism, came from an ancillary study of the original GAIT (Glucosamine/ chondroitin Arthritis Intervention Trial), the aim of which was to determine whether these supplements could treat the pain of knee osteoarthritis. In 2006, researchers found that a subgroup of study participants did show significant pain relief. Researchers said that despite the new study’s limitations, it provided them with insights into several aspects of osteoarthritis, a disease that affects nearly 21 million Americans.—

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